Summary of cGVHD pivotal trials

Drug/pivotal study designIbrutinib phase 1b/2, open-label study5 Ruxolitinib phase 3 open-label, randomized trial (REACH3)6 Belumosudil phase 2 randomized, multicenter registration (ROCKstar Study)7 Axatilimab phase 2, multinational, pivotal, randomized study (AGAVE-201)8 Rovadicitinib multicenter, open-label, phase 1b/2a
Target and mechanism of action BTK inhibitor JAK1/2 inhibitor ROCK2 inhibitor CSF1R blocking antibody Dual JAK1/2 and ROCK1/2 inhibitor 
Reduced activation, proliferation, and survival of B and T cells Reduced inflammation, increased TREGs, reduced collagen deposition Reduced type 17 and follicular T-helper cells and enhanced regulatory T cells Inhibition of monocyte-driven inflammation and fibrosis Combined inhibition of inflammatory and profibrotic pathways 
Study population N = 42 N = 329 N = 132 N = 241 N = 44 
Age 18 years Age > 12 years Age > 12 years Age > 2 years Age > 12 years 
1-3 lines of therapy cGVHD 1 prior line 2-5 prior lines of therapy ≥2 lines of therapy ≥1 prior lines of therapy 
Steroid-refractory: 14%
Steroid-dependent: 67% 
Steroid-refractory: 71.4%
Steroid-dependent: 28.6% 
Refractory to prior line treatment: 72% Refractory to last line of treatment: 48% Steroid-refractory: 25%
Steroid-dependent: 75% 
Lung involvement: 5% Lung involvement: 44.8%
Lung score 3: 8.5%: 
Lung involvement: 36%
Lung score 3: excluded 
Lung involvement: 40% Lung involvement: 59.1%
Lung score 3: 23.1% 
  Prior rux: 29%
Prior ibru: 34% 
Prior rux: 74%
Prior belu: 23% 
Prior rux: 40.9%
Prior belu: 0 
Short-term efficacy BOR (at any time): 67% BOR until week 24: 76.4% BOR (at any time): 76% ORR in first 6 cycles: 74% BOR until week 24: 86.4% 
Long-term efficacy Sustained response beyond 44 weeks: 55% Median FFS above 18.6 mo 12-mo FFS: 57% 12-mo FFS: 64% 12-mo FFS: 85.2% 
AEs leading to discontinuation 33% 16.4% 21% 6%  3.5%  
Drug/pivotal study designIbrutinib phase 1b/2, open-label study5 Ruxolitinib phase 3 open-label, randomized trial (REACH3)6 Belumosudil phase 2 randomized, multicenter registration (ROCKstar Study)7 Axatilimab phase 2, multinational, pivotal, randomized study (AGAVE-201)8 Rovadicitinib multicenter, open-label, phase 1b/2a
Target and mechanism of action BTK inhibitor JAK1/2 inhibitor ROCK2 inhibitor CSF1R blocking antibody Dual JAK1/2 and ROCK1/2 inhibitor 
Reduced activation, proliferation, and survival of B and T cells Reduced inflammation, increased TREGs, reduced collagen deposition Reduced type 17 and follicular T-helper cells and enhanced regulatory T cells Inhibition of monocyte-driven inflammation and fibrosis Combined inhibition of inflammatory and profibrotic pathways 
Study population N = 42 N = 329 N = 132 N = 241 N = 44 
Age 18 years Age > 12 years Age > 12 years Age > 2 years Age > 12 years 
1-3 lines of therapy cGVHD 1 prior line 2-5 prior lines of therapy ≥2 lines of therapy ≥1 prior lines of therapy 
Steroid-refractory: 14%
Steroid-dependent: 67% 
Steroid-refractory: 71.4%
Steroid-dependent: 28.6% 
Refractory to prior line treatment: 72% Refractory to last line of treatment: 48% Steroid-refractory: 25%
Steroid-dependent: 75% 
Lung involvement: 5% Lung involvement: 44.8%
Lung score 3: 8.5%: 
Lung involvement: 36%
Lung score 3: excluded 
Lung involvement: 40% Lung involvement: 59.1%
Lung score 3: 23.1% 
  Prior rux: 29%
Prior ibru: 34% 
Prior rux: 74%
Prior belu: 23% 
Prior rux: 40.9%
Prior belu: 0 
Short-term efficacy BOR (at any time): 67% BOR until week 24: 76.4% BOR (at any time): 76% ORR in first 6 cycles: 74% BOR until week 24: 86.4% 
Long-term efficacy Sustained response beyond 44 weeks: 55% Median FFS above 18.6 mo 12-mo FFS: 57% 12-mo FFS: 64% 12-mo FFS: 85.2% 
AEs leading to discontinuation 33% 16.4% 21% 6%  3.5%  

belu, belumosudil; CSF1R, colony-stimulating factor 1 receptor; FFS, failure-free survival; Ibru, ibrutinib); JAK, Janus kinase; ORR, overall response; ROCK, rho-associated coiled-coil-containing protein kinase; rux, ruxolitinib; TREGs, regulatory T cells.

Data for patients treated with recommended or approved dose.

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